ADVANCE+: A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

• Sponsor: argenx (Industry)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT04225156
• Collaborator: (none)
• Enrollment: 101
• Locations: 82
• Arm: 1
• Anticipated Duration (Months): 65
• Patients Per Site: 1.2
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.

Condition or Disease	Intervention/Treatment	Phase
Primary Immune Thrombocytopenia	Biological: efgartigimod	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 101 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia.
• Actual Study Start Date: Jun 2, 2020
• Anticipated Primary Completion Date: Jul 1, 2025
• Anticipated Study Completion Date: Nov 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: efgartigimod patients receiving efgartigimod	Biological: efgartigimod; Intravenous infusion of efgartigimod; Other Names: ARGX-113

Outcome Measures

Primary Outcome Measures

1. Frequency and severity of Adverse Events [Up to 60 weeks]

2. Frequency and severity of vital signs [Up to 60 weeks]

3. Frequency and severity of laboratory assessments [Up to 60 weeks]

Secondary Outcome Measures

1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L. [Over the 52 weeks of treatment]

2. Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period. [Over the 52 weeks of treatment]

3. Mean change from baseline in platelet count at each visit. [Up to 60 weeks, at each visit]

4. For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L [Up to 60 weeks, at each visit]

5. The percentage of weeks in the trial with platelet counts of ≥30×109/L and at least 20×10E9/L above baseline. [Over the 52 weeks of treatment]

6. In patients with baseline platelet count of <15×10E9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and at least 20×10E9/L above baseline. [Over the 52 weeks of treatment]

7. In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial. [Up to 5 weeks, between visit 19 and 24 of the trial]

8. In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial. [Up to 7 weeks, between visit 17 and 24 of the trial]

9. Rate of receipt of rescue therapy (rescue per patient per month). [Up to 60 weeks, at each visit]

10. Reduction in concurrent ITP therapy. [Up to 60 weeks, at each visit]

11. Incidence and severity of the WHO-classified bleeding events. [Up to 60 weeks, at each visit]

12. Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits. [Up to 52 weeks]

13. Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits. [Up to 52 weeks]

14. Change from baseline in Quality of Life (SF-36) at planned visits. [Up to 52 weeks]

15. Incidence of anti-drug antibodies (ADA) to efgartigimod. [Up to 216 weeks]

16. Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough). [Up to 60 weeks]

17. Pharmacodynamics markers: total IgG. [Up to 60 weeks]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion criteria:

1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).

2. Patients enrolled in the ARGX-113-1801 trial who completed the 24-weeks trial period.

3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. Women are considered of childbearing potential unless they are postmenopausal (defined by continuous amenorrhea) for at least 1 year with a follicle-stimulating hormone (FSH) of >40 IU/L or are surgically sterilized (ie, women who had a hysterectomy, a bilateral salpingectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm postmenopausal status in amenorrheic patients not on hormonal replacement therapy.

4. Women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the

IMP. They must be on a stable regimen, for at least 1 month:

• combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation:

• oral

• intravaginal

• transdermal

• progestogen-only hormonal contraception associated with inhibition of ovulation:

• oral

• injectable

• implantable

• intrauterine device (IUD)

• intrauterine hormone-releasing system

• bilateral tubal occlusion

• vasectomized partner (provided that the partner is the sole sexual partner of the trial participant and that aspermia was documented post-procedure)

• continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not acceptable

• male or female condom with or without spermicide

• cap, diaphragm, or sponge with spermicide.

1. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use an acceptable method of contraception, ie, a condom. Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant) can be included. Sterilized male patients who have had a vasectomy with documented aspermia post-procedure can be included. In addition, male patients are not allowed to donate sperm during this period from signing of informed consent form, throughout the duration of the trial, and for 90 days after the last administration of IMP. In addition to the above criteria, for patients who want to continue receiving efgartigimod during an additional 52-week treatment period (only applicable in case efgartigimod is not yet commercially available for patients with primary ITP, or becomes available through another patient program for patients with primary ITP)

2. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).

3. Patient has completed a 52-week treatment period.

Exclusion criteria:

1. Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).

2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.

3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.

4. Use of any other investigational drug or participation in any other investigational trial.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Investigator Site 0010045	Washington	District of Columbia	United States	20007
2	Investigator Site 0010037	Ocala	Florida	United States	34474
3	Investigator Site 0010042	Iowa City	Iowa	United States	52242
4	Investigator Site 0010040	Columbus	Ohio	United States	43210
5	Investigator Site 0430002	Vienna		Austria
6	Investigator Site 0430003	Vienna		Austria
7	Investigator Site 0320012	Brasschaat		Belgium
8	Investigator Site 0320011	Brugge		Belgium
9	Investigator Site 0320014	Turnhout		Belgium
10	Investigator Site 0320002	Yvoir		Belgium
11	Investigator Site 3590001	Pleven		Bulgaria
12	Investigator Site 3590002	Sofia		Bulgaria
13	Investigator Site 4200001	Brno		Czechia
14	Investigator Site 4200008	Olomouc		Czechia
15	Investigator Site 4200006	Ostrava		Czechia
16	Investigator Site 4200007	Praha		Czechia
17	Investigator Site 0330009	Créteil		France
18	Investigator Site 0330018	Montpellier		France
19	Investigator Site 0330008	Pessac		France
20	Investigator Site 0330016	Périgueux		France
21	Investigator Site 0490010	Düsseldorf		Germany
22	Investigator Site 0490008	Essen		Germany
23	Investigator Site 0360004	Budapest		Hungary
24	Investigator Site 0360006	Debrecen		Hungary
25	Investigator Site 0360015	Győr		Hungary
26	Investigator Site 0360010	Nyíregyháza		Hungary
27	Investigator Site 0360014	Szombathely		Hungary
28	Investigator Site 0390014	Milano		Italy
29	Investigator Site 0390020	Monza		Italy
30	Investigator Site 0390015	Novara		Italy
31	Investigator Site 0390010	Ravenna		Italy
32	Investigator Site 0390011	Reggio Calabria		Italy
33	Investigator Site 0390018	Reggio Emilia		Italy
34	Investigator Site 0390019	Rimini		Italy
35	Investigator Site 0390009	Siena		Italy
36	Investigator Site 0390016	Trieste		Italy
37	Investigator Site 0810015	Hirakata		Japan
38	Investigator Site 0810010	Hiroshima		Japan
39	Investigator Site 0810017	Iruma		Japan
40	Investigator Site 0810022	Kashiwa		Japan
41	Investigator Site 0810018	Maebashi		Japan
42	Investigator Site 0810021	Niigata		Japan
43	Investigator Site 0810014	Sapporo		Japan
44	Investigator Site 0810016	Shibukawa		Japan
45	Investigator Site 0810023	Shimotsuke		Japan
46	Investigator Site 0310006	Den Haag		Netherlands
47	Investigator Site 0310005	Rotterdam		Netherlands
48	Investigator Site 0480012	Gdańsk		Poland
49	Investigator Site 0480013	Katowice		Poland
50	Investigator Site 0480014	Lublin		Poland
51	Investigator Site 0480026	Nowy Sącz		Poland
52	Investigator Site 0480011	Łódź		Poland
53	Investigator Site 0070006	Kaluga		Russian Federation
54	Investigator Site 0070008	Moscow		Russian Federation
55	Investigator Site 0070007	Petrozavodsk		Russian Federation
56	Investigator Site 0070013	Rostov-on-Don		Russian Federation
57	Investigator Site 0070015	Syktyvkar		Russian Federation
58	Investigator Site 0070012	Tula		Russian Federation
59	Investigator Site 0070010	Ufa		Russian Federation
60	Investigator Site 0340006	Barcelona		Spain
61	Investigator Site 0340007	Barcelona		Spain
62	Investigator Site 0340009	Madrid		Spain
63	Investigator Site 0340014	Madrid		Spain
64	Investigator Site 0340012	Palma De Mallorca		Spain
65	Investigator Site 0340015	Pozuelo De Alarcón		Spain
66	Investigator Site 0340013	Sevilla		Spain
67	Investigator Site 0340004	Valencia		Spain
68	Investigator Site 0340011	Valencia		Spain
69	Investigator Site 0900003	Ankara		Turkey
70	Investigator Site 0900006	Ankara		Turkey
71	Investigator Site 0900015	Ankara		Turkey
72	Investigator Site 0900016	Edirne		Turkey
73	Investigator Site 0900013	Istanbul		Turkey
74	Investigator Site 0900004	İzmir		Turkey
75	Investigator Site 0900010	Mersin		Turkey
76	Investigator Site 0900007	Sakarya		Turkey
77	Investigator Site 0900009	Samsun		Turkey
78	Investigator Site 0900017	Tekirdağ		Turkey
79	Investigator Site 0900019	Trabzon		Turkey
80	Investigator Site 3800006	Mykolaiv		Ukraine
81	Investigator Site 0440008	London		United Kingdom
82	Investigator Site 0440012	Southampton		United Kingdom

Sponsors and Collaborators

• argenx

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: argenx
• ClinicalTrials.gov Identifier: NCT04225156
• Other Study ID Numbers: ARGX-113-1803; 2019-002101-21
• First Posted: Jan 13, 2020
• Last Update Posted: Mar 7, 2022
• Last Verified: Mar 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic

Study Results

No Results Posted as of Mar 7, 2022